Secretory diarrhea

The Age for Porosome Therapeutics is Here BOSTON--(BUSINESS WIRE)-- Porosome Therapeutics, Inc. (Porosome Therapeutics) today announced the publishing of a study that brings promise for a cure to cystic fibrosis (CF). The preprint highlights the ground-breaking therapeutic approach that overcomes CF nearly four times more effectively than currently available medications to treat the inherited disorder. “We’re excited about the potential the porosome platform holds for developing new and novel drugs and therapies to address a wide range of secretory disorders,” said Professor Bhanu P. Jena, PhD, Founder and Chairman, Porosome Therapeutics. “Echoing the International Patent Search Agency, our central claims are ‘novel’ and ‘inventive,’” said Guillermo Marmol, President and CEO, Porosome Therapeutics. “We look forward to bringing such new, novel, safe, and curative treatments to market for people living with cystic fibrosis, diabetes, and other secretory disorders.” On December 9, 2023, the preprint entitled “Reprogramming the Cells Secretory Machinery: A Cystic Fibrosis Rescue” was published on bioRxiv. The study identified an innovative therapeutic solution for CF patients by first determining the relationship between the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene and porosome-mediated secretion in human airways. Results demonstrate that mutation in porosome-associated CFTR also affects other proteins within the porosome secretory machine, thereby reducing mucus secretion. Researchers then reconstituted functional porosomes into the plasma membrane of CFTR mutant cells to demonstrate that porosome reconstitution normalizes mucus secretion approximately four times more effectively than currently available CF drugs. “Our company research has aided in the ability to identify the critical role CFTR plays within the porosome secretory machinery. By harnessing this technology, we can advance innovative treatments for currently incurable diseases,” said Marmol. “Our patient-centered approach in drug design and development at Porosome Therapeutics will transform therapeutic options for patients diagnosed with cystic fibrosis, diabetes, cancer, Alzheimer’s, and other neurological, digestive, and immune disorders.” CF is a genetic condition that adversely affects cells in the body that secrete mucus, such as the airways, digestive system, and reproductive organs. Normally, these fluids are slick and thin. However, mutations in the CFTR gene in people with CF make the secretions thick and sticky. The secretions clog tubes, ducts, and passages rather than lubricating them, preventing normal function. While more than 2,000 mutations in the CFTR gene have been identified, the ΔF508 CFTR is the most common, accounting for approximately 70% of all CFTR mutations. It is estimated that more than 160,000 people worldwide are living with CF, with a consequent reduction in life expectancy. With this study, Porosome Therapeutics has demonstrated positive pre-clinical results of porosome-reconstitution therapy in human bronchial air-liquid interface 3D cultures that mimic normal lung physiology, responding to CFTR inhibitors and the CF corrector and modulator drugs tezacaftor and ivacaftor. The company is currently conducting in vivo animal studies. About Porosome Therapeutics, Inc. Porosome Therapeutics, Inc. is a Boston-based biopharmaceutical company utilizing the breakthrough discovery of the porosome, the cell’s secretory machinery, and novel technologies to identify and create proprietary high-value therapeutics for currently undruggable porosome proteins relating to secretory and hydration disorders such as cystic fibrosis, diabetes, and cancer. Porosome Therapeutics is the first company with a technology platform that develops novel therapies targeting the porosome, the universal secretory machinery in cells. This platform is based on decades of research supported by more than 200 publications and is effective against many serious diseases involving secretory defects. The platform also specializes in highly specific nanobody-mediated targeted therapies that greatly reduce drug side effects. View source version on businesswire.com: Contacts Laramie Langdon laramie@perrycom.com (916) 658-0144 Source: Porosome Therapeutics, Inc. View this news release online at:

MVID, for which there are no approved drug treatments, is a life-threatening and ultra-rare autosomal recessive disease that affects newborns and children, leading to intestinal failure, significant morbidity and even death from severe secretory diarrhea Crofelemer has been granted Orphan Drug Designation by the FDA and the European Medicines Agency for MVID, and the Company's Investigational New Drug application for crofelemer for treatment of MVID is now activated by the FDA SAN FRANCISCO, CA / ACCESSWIRE / October 19, 2023 / Jaguar Health, Inc. (NASDAQ:JAGX) ("Jaguar" or the "Company") today announced that an independent, investigator-initiated study evaluating the efficacy of crofelemer, the Company's plant-based prescription drug, for the treatment of microvillus inclusion disease (MVID) has been published in The Journal of Clinical Investigation (JCI), a peer-reviewed medical journal covering biomedical research. The results of this in vitro study demonstrate that crofelemer has the clinical potential for symptomatic therapy and electrolyte and fluid management in MVID patients. Click here to view the published study. MVID is a severe infantile disease characterized by diarrhea, malabsorption, and acid/base instability, requiring intensive parenteral support for nutritional and fluid management, and there are currently no approved drug treatments. This study, titled "Patient-derived enteroids provide a platform for the development of therapeutic approaches in microvillus inclusion disease," was conducted by Jay Thiagarajah, MD, PhD, who is Co-Director of the Congenital Enteropathy Program, and an Attending Physician in the Division of Gastroenterology, Hepatology and Nutrition, at Boston Children's Hospital, Harvard Medical School, where Dr. Thiagarajah also serves as Assistant Professor of Pediatrics. MVID patients commonly face intestinal failure - a catastrophic health situation that also often afflicts patients with short bowel syndrome (SBS). Crofelemer has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for both MVID and SBS. Jaguar is supporting investigator-initiated and investigator IND proof-of-concept studies of crofelemer for MVID and SBS with intestinal failure in the US, EU, and Middle East/North Africa (MENA) regions, with results expected before the end of 2023 and in 2024. In accordance with the guidelines of specific European Union countries, published data from such clinical investigations could support reimbursed early patient access to crofelemer for SBS or MVID, potentially in 2024, for these debilitating conditions. The Company's Investigational New Drug application for crofelemer for the treatment of MVID was activated by the FDA August 7, 2023. About Crofelemer Crofelemer is the only oral prescription drug approved by the FDA under botanical guidance. It is plant-based, extracted and purified from the red bark sap of the Croton lechleri tree in the Amazon Rainforest. Jaguar family company Napo Pharmaceuticals has established a sustainable harvesting program, under fair trade practices, for crofelemer to ensure a high degree of quality, ecological integrity, and support for Indigenous communities. About the Jaguar Health Family of Companies Jaguar Health, Inc. (Jaguar) is a commercial stage pharmaceuticals company focused on developing novel proprietary prescription medicines sustainably derived from plants from rainforest areas for people and animals with gastrointestinal distress, specifically associated with overactive bowel, which includes symptoms such as chronic debilitating diarrhea, urgency, and bowel incontinence. Jaguar family company Napo Pharmaceuticals focuses on developing and commercializing human prescription pharmaceuticals for essential supportive care and management of neglected gastrointestinal symptoms across multiple complicated disease states. Napo Pharmaceuticals' crofelemer drug product candidate is the subject of the OnTarget study, an ongoing pivotal Phase 3 clinical trial for preventive treatment of chemotherapy-induced overactive bowel (CIOB) in adults with cancer on targeted therapy. Jaguar family company Napo Therapeutics is an Italian corporation Jaguar established in Milan, Italy in 2021 focused on expanding crofelemer access in Europe and specifically for orphan and/or rare diseases. Jaguar Animal Health is a Jaguar tradename. Magdalena Biosciences, a joint venture formed by Jaguar and Filament Health Corp. that emerged from Jaguar's Entheogen Therapeutics Initiative (ETI), is focused on developing novel prescription medicines derived from plants for mental health indications. For more information about: Jaguar Health, visit Napo Pharmaceuticals, visit Napo Therapeutics, visit napotherapeutics.com Magdalena Biosciences, visit magdalenabiosciences.com Visit Jaguar on LinkedIn: Visit Jaguar on X: Visit Jaguar on Instagram: Forward-Looking Statements Certain statements in this press release constitute "forward-looking statements." These include statements regarding Jaguar's expectation that results of investigator-initiated and investigator IND proof-of-concept studies of crofelemer for MVID and SBS with intestinal failure will be available before the end of 2023 and in 2024, and Jaguar's expectation that published data from such clinical investigations could support reimbursed early patient access to crofelemer for SBS or MVID, potentially in 2024, for these conditions. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "aim," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential" or "continue" or the negative of these terms or other similar expressions. The forward-looking statements in this release are only predictions. Jaguar has based these forward-looking statements largely on its current expectations and projections about future events. These forward-looking statements speak only as of the date of this release and are subject to a number of risks, uncertainties and assumptions, some of which cannot be predicted or quantified and some of which are beyond Jaguar's control. Except as required by applicable law, Jaguar does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Contact: hello@jaguar.health Jaguar-JAGX SOURCE: Jaguar Health, Inc. View source version on accesswire.com:

MVID is a life-threatening and ultra-rare autosomal recessive disease that affects newborns and children, leading to intestinal failure, significant morbidity and even death from severe secretory diarrhea SAN FRANCISCO, CA / ACCESSWIRE / August 8, 2023 / Napo Pharmaceuticals, Inc. (Napo), a Jaguar Health, Inc. (NASDAQ:JAGX) company, today announced the activation by the U.S. Food and Drug Administration (FDA) of the Investigational New Drug (IND) application for a new crofelemer powder for oral solution formulation for the treatment of microvillus inclusion disease (MVID), an ultra-rare congenital diarrheal disorder (CDD). "We were very pleased to hear that the FDA has communicated that we may proceed with the proposed phase 2 clinical trial for crofelemer for treatment of MVID in pediatric patients with a novel formulation of crofelemer," said Lisa Conte, Napo and Jaguar's President and CEO. "MVID is a life-threatening and rare autosomal recessive disease that affects newborns and children, leading to intestinal failure, significant morbidity, and even death from severe secretory diarrhea. While there are currently no approved therapeutic treatments for MVID, parenteral nutrition (PN), the standard of care for management of MVID, can cost up to $150,000 a year or more with complications. MVID patients suffer from severe cholera-like diarrhea, and symptomatic management of diarrhea in MVID may reduce their dependence on PN. We plan to host an investor-facing webinar in the near future with leading pediatric gastroenterologists to further elaborate the value of managing diarrhea in MVID patients with intestinal failure." Crofelemer has been granted Orphan Drug Designation (ODD) by the FDA and the European Medicines Agency (EMA) for MVID. The ODD program in both the US and European Union qualifies sponsors to receive potential incentives to develop therapies for the diagnosis, prevention, or treatment of rare diseases or conditions. Crofelemer has also been granted ODD by the FDA and the EMA for short bowel syndrome (SBS). SBS patients with intestinal failure are also treated with PN. Jaguar is supporting investigator-initiated proof-of-concept studies of crofelemer for SBS with intestinal failure and MVID in the EU and Middle East/North Africa (MENA) regions. In accordance with the guidelines of specific EU countries, published data from such clinical investigations could support reimbursed early patient access to crofelemer for SBS or MVID, potentially in 2024, for these debilitating conditions. About Crofelemer Crofelemer is the only oral FDA approved drug under botanical guidance. It is plant-based, extracted and purified from the red bark sap of the Croton lechleri tree in the Amazon Rainforest. Napo Pharmaceuticals has established a sustainable harvesting program, under fair trade practices, for crofelemer to ensure a high degree of quality, ecological integrity, and support for indigenous communities. About Jaguar Health, Napo Pharmaceuticals & Napo Therapeutics S.p.A Napo Pharmaceuticals, a Jaguar Health company, focuses on developing and commercializing proprietary human pharmaceuticals from plants harvested responsibly from rainforest areas. Jaguar Health is the majority shareholder of Napo Therapeutics S.p.A. (f/k/a Napo EU S.p.A.), an Italian corporation established by Jaguar Health in Milan, Italy in 2021 that focuses on expanding crofelemer access in Europe. For more information about Jaguar Health, please visit . For more information about Napo Pharmaceuticals, visit . For more information about Napo Therapeutics, visit napotherapeutics.com. Forward-Looking Statements Certain statements in this press release constitute "forward-looking statements." These include statements regarding the expectation that an investor-facing webinar will be held in the near future with leading pediatric gastroenterologists to further elaborate the value of managing diarrhea in MVID patients with intestinal failure, and the expectation that published data from proof-of-concept studies could support reimbursed early patient access to crofelemer for SBS or MVID with intestinal failure, potentially in 2024. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "aim," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential" or "continue" or the negative of these terms or other similar expressions. The forward-looking statements in this release are only predictions. Jaguar has based these forward-looking statements largely on its current expectations and projections about future events. These forward-looking statements speak only as of the date of this release and are subject to a number of risks, uncertainties and assumptions, some of which cannot be predicted or quantified and some of which are beyond Jaguar's control. Except as required by applicable law, Jaguar does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Contact: hello@jaguar.health Jaguar-JAGX SOURCE: Jaguar Health, Inc. View source version on accesswire.com: